A Global Study of Haematopoietic Stem Cell Transplantation in Multiple Sclerosis and Other Neurodegenerative Disorders

Aim: The aim of this research is to understand the uptake of haemopoietic stem cell transplantation (HSCT) in neuroimmunological disorders like multiple sclerosis (MS). Methods: A global internet survey was conducted of people having had HSCT, comparing demographics, treatment protocol, and effectiveness. Results: Of 271 participants useful data was available in 223. 73.5% were female in the age range of 35-54 years, most had a household income greater than US$50,000 and the majority of participants were from Australia and the United States of America. 94.6% had MS. Most had their treatment in Russia (38.7%) and 78.1% had non-myeloablative transplants. Nearly half of the participants spent between US$50,000 to US$74,999. 54.5% of neurologists were not supportive of their patients having HSCT. 85.5% of participants believed HSCT helped them manage their disease from weeks to years after transplantation. 9.45% would recommend the treatment. The average reduction in Expanded Disability Status Score after transplantation was 1.2 [95% CI; 0.97-1.41; N=197; p < 0.0001; t: 10.7, df 196]. Conclusions: Participants were supportive of HSCT despite the costs and would recommend it to others. The data suggest some benefit in minimizing disability in MS and provides justification for large randomized controlled trials.

Clinical management of sickle cell liver disease in children and young adults

Liver involvement in sickle cell disease (SCD) is often referred to as sickle cell hepatopathy (SCH) and is a complication of SCD which may be associated with significant mortality. This review is based on a round-table workshop between paediatric and adult hepatologists and haematologists and review of the literature. The discussion was prompted by the lack of substantial data and guidance in managing these sometimes very challenging cases. This review provides a structured approach for the diagnosis and management of SCH in children and young adults. The term SCH describes any hepatobiliary dysfunction in the context of SCD. Diagnosis and management of biliary complications, acute hepatic crisis, acute hepatic sequestration and other manifestations of SCH are discussed, as well as the role of liver transplantation and haemopoietic stem cell transplantation in the management of SCH.

Lymphoid and haematological malignancy and related conditions

Chapter 8 discusses lymphoid and haematological malignancy and related conditions in children and young people. Radiotherapy is less frequently used in haematological malignancy and allied disorders than it once was. In Hodgkin lymphoma, better risk stratification, more effective systemic therapy and the use of 18F-fluoro-deoxyglucose positron emission tomography (18F-FDG-PET) imaging to assess response to chemotherapy have facilitated a safe reduction in the indications for radiotherapy. Nonetheless, radiotherapy remains an important component of multimodality therapy in patients with advanced or poorly responding disease. In acute leukaemia, radiotherapy is no longer used as part of first-line treatment schedules. Instead, it is reserved for refractory and relapsed disease. Total body irradiation remains valuable as part of conditioning prior to haemopoietic stem cell transplantation. Cranial and testicular radiotherapy is helpful for selected patients who relapse in these sanctuary sites. Radiotherapy is also indicated infrequently in some unusual and benign haematological conditions.